N-{(2S,3R)-4,4-DIFLUORO-1-(2-HYDROXY-2-METHYLPROPANOYL)-2-[(2,3',5'-TRIFLUORO[1,1'-BIPHENYL]-3-YL)METHYL]PYRROLIDIN-3-YL}ETHANESULFONAMIDE for Narcolepsy Type 1 (Narcolepsy with Cataplexy)

Inclusion criteria

• {"criterion_text":"- The participant has an ICSD-3 or ICSD-3-TR diagnosis of NT1."}
• {"criterion_text":"- Male or female participants aged 6 to 17 years, inclusive, at the time of signing the ICF."}
• {"criterion_text":"- The participant has a body mass index within the range 18 to 30 kg/m2"}
• {"criterion_text":"- The participant is positive for the human leukocyte antigen (HLA) genotype HLA-DQB1*06:02 or results from radioimmunoassay indicate the participant’s CSF OX/hypocretin-1 concentration is <110…pg/mL"}
• {"criterion_text":"- The participant is judged by the investigator to be sufficiently healthy to participate in the trial based on clinical evaluations including laboratory safety tests, medical history, physical examination, 12-lead ECG, and vital sign measurements performed at the screening visit and before the first dose of trial intervention."}
• {"criterion_text":"- Parent or legal guardian – and participants old enough to understand - have expressed a willingness to participate in and complete the trial and signed and dated the ICF after getting acquainted with the participant’s information sheet, prior to beginning protocol-required procedures."}

Exclusion criteria

• {"criterion_text":"- Participant is unable to refrain from using excluded medications"}
• {"criterion_text":"- The participant has a positive urine screen result for drugs of abuse and/or positive alcohol test result at screening or at Day -1."}
• {"criterion_text":"- Participant has a positive pregnancy test result at screening or is breastfeeding."}
• {"criterion_text":"- Participant has a known hypersensitivity to any component of the formulation of oveporexton or related compounds."}

Primary endpoints

• {"endpoint_text":"- Maximum observed concentration (Cmax). Time to reach maximum observed concentration (tmax). Area under the concentration-time curve from time 0 to infinity (AUC∞), calculated using the observed value of the last quantifiable concentration.","definition_or_measurement_approach":"Plasma pharmacokinetic (PK) parameters measured in plasma: Cmax, Tmax, and AUC∞ calculated using observed last quantifiable concentration."}

Secondary endpoints

• {"endpoint_text":"- Occurrence of at least 1 TEAE.","definition_or_measurement_approach":"Safety assessed by incidence of treatment-emergent adverse events (TEAEs)."}
• {"endpoint_text":"- Participants rating acceptability and palatability.","definition_or_measurement_approach":"Acceptability/palatability assessed by the Acceptability/Palatability Questionnaire."}

Italy

Earliest CTIS Part Ii Submission Date

03-11-2025

Latest Decision Or Authorization Date

27-11-2025

Processing Time Days

24

Number Of Sites

1

Number Of Participants

16

Primary sponsor

Full Name

Takeda Development Center Americas Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Signant Health Global LLC

Responsibilities

eCTP

Name

Cogstate Limited

Responsibilities

Cognitive Testing

Name

PPD Development LP

Responsibilities

PK

Name

Eresearchtechnology Inc.

Responsibilities

ECG analysis/review

Name

PPD Global Central Labs

Responsibilities

Safety labs

Name

Pharmaceutical Product Development LLC

Third parties

• {"country":"United States","full_name":"Signant Health Global LLC","duties_or_roles":"eCTP","organisation_type":"Pharmaceutical company"}
• {"country":"Australia","full_name":"Cogstate Limited","duties_or_roles":"Cognitive Testing","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"PK","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ECG analysis/review","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"Safety labs","organisation_type":"Pharmaceutical company"}